INTRODUCTIONThe service oriented and dynamic nature of the healthcare industry has led to the discovery of a plethora of treatment options for a wide range of ailments. However, there are certain diseases, termed as rare, that continue to remain untreated and neglected. Rare / orphan diseases, as the name suggests, are those diseases which are relatively infrequent in occurrence. Many orphan diseases are life-threatening and progressive in nature. Absence of potential treatment regimes catering to these diseases is a big unmet need in the market.

The prevalence and adversity of these ailments has gained significant attention in the global healthcare industry. Stakeholders have realized the necessity and importance of urgently addressing the widening concerns and the need for discovering treatment options for several rare diseases. There is a tremendous scope offered by the innovation driven research being executed by various organizations in an effort to bridge gaps in the current market landscape.

The enactment of the Orphan Drugs Act in the US and EU emerged as ground breaking and provided the necessary support to guide research focused on rare diseases worldwide. The orphan designated drugs are granted important incentives which include market exclusivity and fees reductions. This regulatory assistance has provided a strong impetus to the pharmaceutical companies and led to their active participation in the market. Collaborative agreements and partnerships, amongst the pharmaceutical firms and other active organizations (such as EURORDIS, NORD and Global Genes), are also playing a key role in addressing the needs of this market. The wide gap between the demand and supply of drugs is likely to provide a major boost to the market in the coming decade. Several drugs for various rare diseases are currently under development and, once approved, are likely to be readily accepted by the untreated patient population.

SCOPE OF THE REPORTThe ‘Rare Diseases: The Pharmaceutical Landscape and Opportunity Areas’ report is a comprehensive study of the market of drugs being developed by the top pharmaceutical companies for rare diseases. The focus of this study is primarily to uncover several initiatives currently underway to harness the potential presented in different therapeutic areas. One of the basic building blocks for analysing the market was to prepare an extensive pipeline of drugs for rare diseases. This effort encompassed reviewing several databases, company websites and other information available in the public domain.

We confined the focus of our study to 50 large pharmaceutical companies which invest a proportion of their R&D spend on drugs for rare diseases. These pharmaceutical companies were profiled to get an in-depth view of the work being done by them in this domain. The profiles include a general overview of the firms, financial performance over the last five to six years, and the current marketed and clinical pipeline products specific to rare diseases.

With a robust pipeline in place, we then analysed where the industry’s current vision lies and highlighted a number of efforts which have been taken by pharmaceutical companies, healthcare organizations and other industry stakeholders. Specifically, our market potential analysis outlines key patient demographics and underscores potential areas anticipated to emerge as major revenue generators for the pharmaceutical companies. In addition to the above, we have also provided our own independent perspective on the key drivers behind the growth of this market, and the associated challenges.

RESEARCH METHODOLOGYMost of the data presented in this report has been gathered via secondary and primary research. For most of our projects, we conduct interviews with experts in the area (academia, industry, medical practice and other associations) to solicit their opinions on emerging trends in the market. This is primarily useful for us to draw out our own opinion on how the market will evolve across different regions and technology segments. Where possible, the available data has been checked for accuracy from multiple sources of information.

CHAPTER OUTLINESChapter 2 provides a general introduction to rare diseases. We have highlighted the indispensable role played by several regulatory organizations in accelerating this market. The chapter also elaborates upon the various drivers responsible for the growth in this market.

Chapter 3 provides a comprehensive market landscape of drugs in development for rare diseases. This chapter includes an elaborate analysis of pipeline drugs which are currently in different stages of development. The detailed analysis includes information on the targeted indications, types of molecules and the current phases of development. The chapter also provides the historic ladder for the orphan drug designations conferred to several drugs in the US and the European markets.

Chapter 4 offers an insightful discussion of six most popular indications targeted by the drugs under development. This analysis revolves around current landscape, prevailing competition, anticipated shift in the market trend and the key focus for the future.

Chapter 5 provides detailed company profiles of the top pharmaceutical companies focusing in the field of rare diseases. Each company profile includes information such as financial performance, geographical presence, marketed / pipeline drugs, recent collaborations specific to rare diseases and the initiatives taken by the firm towards the treatment of rare diseases.

Chapter 6 summarises the overall report. In this chapter, we have provided a recap of the key takeaways and our overall opinion on where the market is headed in the mid-long term.

Chapter 7 provides the list of companies covered in this report.

EXAMPLE HIGHLIGHTS1. With over 7000 rare diseases reported in the world, lack of awareness, dispersed target population and inefficient reporting of these diseases limits the pace of development in this industry. However, the industry-academia coalition has helped in filling some of these gaps. An active participation by pharma firms through various initiatives, such as the Rare Disease Day, has helped to address the existing lacunae in the demand supply gap for these drugs.2. The market is characterized by the presence of several pipeline drugs under development for the treatment of rare diseases. Of these, 30% drugs are in phase III of clinical development whereas 38% of drugs are under development in phase II clinical trials. Phase I drugs account for 20% to the total market. 3. Companies such as Roche, BMS, Sanofi, GSK, Boehringer Ingelheim, Celgene, Novartis and Pfizer are some of the key players in the market. However, the market remains opportunistic for newer players to step in and benefit from the vast potential in different therapeutic areas.4. With a share of more than 50%, oncology remains the key focus area of pharmaceutical firms. Blood associated diseases and neural disorders are some other well-known areas being targeted.5. On an average, 13-14 drugs are being developed by each pharmaceutical company covered in our analysis. However, there is a stark variation amongst the companies. Some of the bigger pharma companies, such as Roche and BMS, have a much wider portfolio.

6. CONCLUSION6.1. The Emerging Focus in a Widely Untapped Market6.2. Several Stakeholders have Collaborated to Lead the Initiatives6.3. Big Pharma are the Major Contributors6.4. Industry- Academia Coalition: Expunge the Market Constraints6.5. Rare Disease Market: Overall Opportunity is Big

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